Safety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec in Patients With Hemophilia A or B Respectively
- Conditions
- Hemophilia BHemophilia A
- Interventions
- Diagnostic Test: Testing of hepatic AAV Vector integration
- Registration Number
- NCT05568719
- Lead Sponsor
- Pfizer
- Brief Summary
A study to learn about the long-term safety and efficacy of giroctocogene fitelparvovec or fidanacogene elaparvovec in patients with hemophilia A or hemophilia B respectively, who have received treatment through prior participation in a Pfizer-sponsored clinical trial. Data collection and participant visits will be based on standard of care.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- Male
- Target Recruitment
- 263
-Only participants who received investigational giroctocogene fitelparvovec or fidanacogene eleparvovec and were enrolled in a Pfizer-sponsored study (C0371002, C0371003, C0371005, C3731001, C3731003) are eligible.
-None
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Hemophilia B / fidanacogene elaparvovec Testing of hepatic AAV Vector integration Participants have received treatment with fidanacogene elaparvovec in a previous study and are not receiving any investigational product in this study Hemophilia A / giroctocogene fitelparvovec Testing of hepatic AAV Vector integration Participants have received treatment with giroctocogene fitelparvovec in a previous study and are not receiving any investigational product in this study
- Primary Outcome Measures
Name Time Method Incidence of factor inhibitor development Day 1 to 10 years FIX inhibitor development was defined as an inhibitor titer \>= 0.6 Bethesda units per milliliter (BU/mL).
Incidence of liver abnormalities Day 1 to 10 years Factor activity level Day 1 to 10 years Factor activity level will be reported. Factor levels may be measured using different assay methods including a one-stage assay or by chromogenic substrate assay and a second one-stage assay.
Incidence of thromboembolic events Day 1 to 10 years Incidence of hepatic malignancy Day 1 to 10 years
- Secondary Outcome Measures
Name Time Method Consumption of exogenous factor (excluding infusions related to surgery) Day 1 to 10 years The annualized TFC in international units (IU) will be derived for each participant for each observation period using the following formula:
Annualized TFC = (Total units of FIX infused (IU)/ Days in observation period) x 365.25 days/yearIncidence of Non-hepatic malignancy Day 1 to 10 years All cause mortality Day 1 to 10 years All-cause mortality was defined as the death due to any cause during the course of study. Incidence rate was defined as the total number of participants with admissible events divided by the total (for all qualifying participants) time at risk for the cohort/treatment group of interest. Incidence rate of all-cause deaths was reported in this outcome measure.
AIR of exogenous factor (excluding infusions related to surgery) Day 1 to 10 years The AIR or the annualized number of FIX infusions per year, will be derived for each participant for each observation period by using the following formula:
AIR = (Number of FIX infusions / Days in observation period) x 365.25 days/year.Incidence of Auto-immune disorders Day 1 to 10 years Incidence of and time from vector infusion to resumption of prophylaxis Day 1 to 10 years Describe incidence of resumption of prophylaxis resumption and the time (in days) to resumption of prophylaxis after receiving vector infusion.
EQ-5D-5L dimension and VAS scores Day 1 to 10 years The EQ-5D-5L comprises a 5-item health status measure and a visual analog rating scale/feeling thermometer. Using the 5-dimensional Health State Classification, participants are asked to respond to five questions on different aspects of their health status that assess the following:
1. Mobility
2. Self-care
3. Usual activities
4. Pain/Discomfort
5. Anxiety/DepressionTotal ABR (treated or untreated; (excluding bleeds related to surgery) Day 1 to 10 years ABR (Annual Bleed Rate): number of bleeding episodes per year. This includes treated and untreated bleeds.
The ABR or the annualized number of bleeding episodes per year, will be derived for each participant for each observation period by using the following formula:
ABR = (Number of bleeds / Days in observation period) x 365.25 days/year.Incidence of SAEs Day 1 to 10 years An SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly; development of a clinical thrombotic event; development of factor inhibitor; development of a hepatic malignancy; development of drug-related elevated hepatic transaminases that fail to improve with immunosuppressive regimens; occurrence of a malignancy with reasonable possibility of being related to study drug.
Trial Locations
- Locations (11)
UC Davis Health
🇺🇸Sacramento, California, United States
UC Davis Ambulatory Care Clinic
🇺🇸Sacramento, California, United States
UC Davis Hemophilia Treatment Center
🇺🇸Sacramento, California, United States
UC Davis Medical Center
🇺🇸Sacramento, California, United States
UCSF Outpatient Hematology Clinic
🇺🇸San Francisco, California, United States
USF Health Morsani Center For Advanced Healthcare
🇺🇸Tampa, Florida, United States
Mississippi Center For Advanced Medicine
🇺🇸Madison, Mississippi, United States
The Childrens Hospital of Philadelphia Division of Hematology
🇺🇸Philadelphia, Pennsylvania, United States
Washington Institute for Coagulation
🇺🇸Seattle, Washington, United States
Royal Prince Alfred Hospital
🇦🇺Camperdown, New South Wales, Australia
Ege Universitesi Hastanesi
🇹🇷İzmir, İ̇zmir, Turkey